Disease The intractable diseases designated by MHLW, Japan
Diseases : 338 - Clinical trials : 33,695 / Drugs : 21,110 - ( DrugBank : 2,155 ) / Drug target genes : 623 - Drug target pathways : 291
Showing 1 to 10 of 338 diseases
| ID | Disease name [Group] | Clinical trial Phase 1 / 2 / 3 / 4 | Drug [ DrugBank ] | Target gene Target pathway | Domestic patients (1) MHLW, (2) Med expenses recipients (FY2021) |
|---|---|---|---|---|---|
| 1 | Spinal and bulbar muscular atrophy [Neu] 💬 "Spinobulbar muscular atrophy", "SBMA", "Kennedy disease", "Kennedy-Alter-Sung syndrome" | 17 17 trials 0 / 9 / 2 / 1 💬 | 16 16 drugs [ 8 8 drugs ] | 10 10 genes 17 pathways | 1641 (1) 1,223 patients (2) 1,641 patients Age distribution  💬 |
| 2 | Amyotrophic lateral sclerosis [Neu] 💬 "ALS" | 624 624 trials 230 / 280 / 220 / 27 💬 | 611 611 drugs [ 160 160 drugs ] | 172 172 genes 225 pathways | 9968 (1) 9,096 patients (2) 9,968 patients Age distribution  💬 |
| 3 | Spinal muscular atrophy [Neu] 💬 "Myelopathic muscular atrophy", "Spinal muscular atrophy type I", "SMA I", "Werdnig-Hoffman disease" ... | 217 217 trials 112 / 116 / 102 / 27 💬 | 149 149 drugs [ 33 33 drugs ] | 54 54 genes 80 pathways | 929 (1) 712 patients (2) 929 patients Age distribution  💬 |
| 4 | Primary lateral sclerosis [Neu] 💬 "PLS" | 7 7 trials 3 / 2 / 0 / 1 💬 | 17 17 drugs [ 9 9 drugs ] | 19 19 genes 32 pathways | 140 (1) 175 patients (By the research group) (2) 140 patients Age distribution  💬 |
| 5 | Progressive supranuclear palsy [Neu] 💬 "PSP" | 95 95 trials 46 / 43 / 11 / 6 💬 | 119 119 drugs [ 40 40 drugs ] | 65 65 genes 108 pathways | 12557 (1) About 8,100 patients (2) 12,557 patients Age distribution  💬 |
| 6 | Parkinson disease [Neu] 💬 "Disease Parkinson's" | 2,298 2,298 trials 830 / 689 / 593 / 293 💬 | 2,202 2,202 drugs [ 350 350 drugs ] | 188 188 genes 202 pathways | 140473 (1) About 108,800 patients (estimated from Parkinson disease related diseases) (2) 140,473 patients Age distribution  💬 |
| 7 | Corticobasal degeneration [Neu] 💬 "Corticobasal syndrome", "CBD" | 18 18 trials 5 / 3 / 1 / 0 💬 | 35 35 drugs [ 13 13 drugs ] | 9 9 genes 38 pathways | 4517 (1) 3,500 patients (2) 4,517 patients Age distribution  💬 |
| 8 | Huntington disease [Neu] 💬 "Huntington chorea" | 229 229 trials 119 / 123 / 50 / 16 💬 | 193 193 drugs [ 60 60 drugs ] | 84 84 genes 158 pathways | 918 (1) 933 patients (2) 918 patients Age distribution  💬 |
| 9 | Neuroacanthocytosis [Neu] 💬 "Choreoacanthocytosis", "Chorea-acanthocytosis", "Levine-Critchley syndrome", "McLeod syndrome", "Hu ... | 0 - | 0 - | 0 - | 35 (1) Less than 100 patients (By the research group) (2) 35 patients Age distribution  💬 |
| 10 | Charcot-Marie-Tooth disease [Neu] 💬 "CMT", "Charcot-Marie-Tooth disease type 1", "CMT1", "Demyelinating CMT", "Charcot-Marie-Tooth disea ... | 39 39 trials 23 / 16 / 24 / 4 💬 | 44 44 drugs [ 9 9 drugs ] | 11 11 genes 15 pathways | 781 (1) 6,250 patients (By the research group) (2) 781 patients Age distribution  💬 |